Highlighted Publications
Mapping the cellular origin and early evolution of leukemia in Down syndrome
Science, Jul 09, 2021
We utilized CRISPR/Cas9-mediated genome editing in human primary disomic and trisomic hematopoietic stem cells to generate an in vivo model of Down syndrome associated leukemia evolution. This humanized model enabled us to study the mechanism of predisposition, preleukemia initiation and leukemic transformation in order to identify potential therapeutic targets of the disease.
Functional profiling of single CRISPR/Cas9-edited human long-term hematopoietic stem cells
Nat Communications, Oct 18, 2019
We established a state-of-the-art electroporation-based approach of CRISPR/Cas9-mediated genome editing for human primary long-term hematopoietic stem cells. Our methodology allows rapid interrogation of gene deletions, splice variants and fusion genes in order to study gene function relationships in normal and malignant blood stem cells.
